Multiple Myeloma Research Foundation (MMRF) Research Collaboration
Advances Most Comprehensive Characterization of Myeloma Genome to Date
in Esteemed Scientific Journal

NORWALK, Conn.--(BUSINESS WIRE)--The Multiple Myeloma Research Foundation (MMRF) today hailed a new
publication in Cancer Cell as a major step forward in the
organization’s efforts to advance precision medicine in the treatment of
myeloma through the most comprehensive characterization of the myeloma
genome to date. Researchers from the Broad Institute, the Translational
Genomics Research Institute, the Multiple Myeloma Research Consortium
(MMRC) and the MMRF today published an analysis of 203 multiple myeloma
genome sequences, expanding upon the original set of 38 published in Nature
in 2011.

The data identified significantly mutated genes, genetic alterations and
pathways that may respond to molecularly-directed therapies or drug
combinations and pave the way to precision medicine. In this analysis,
specifically, researchers were able to identify new genetic lesions that
had not been observed in the original set of 38 sequences, which, at the
time, had been the largest set of whole genome sequences published in
any cancer. Most excitingly for patients, some of the new molecular
alterations identified may be amenable to near-term precision trials
because there are FDA-approved treatments for other types of cancer that
could be repurposed for myeloma. Additionally, the findings provided
further evidence that genetically distinct clones of myeloma exist
within an individual patient, an observation that will have important
clinical implications.

“Today’s publication would not have been possible without extraordinary
partners working side by side with us over the last eight years to
enable the tissue collection and processing as well as molecular
profiling and data analysis,” said Walter Capone, President of the MMRF.
“We look forward to continuing our collaboration with these and other
world-class centers to conduct clinical trials in molecularly-defined
subtypes of patients, which will accelerate precision medicine and a
cure for multiple myeloma. A network of many motivated centers working
together is critical to accomplish such an effort in an uncommon and
heterogeneous cancer like multiple myeloma.”

Previous genomic studies using a small number of samples hinted at
clonal diversity in myeloma. This present effort, because of its size
and the high-resolution analytical approaches used, is unprecedented in
its comprehensiveness to precisely assess the clonality within a
patient’s disease and the distribution of identified mutations. For
example, one third of multiple myeloma patients with a BRAF V600E
mutation also harbor a clone with a KRAS or an NRAS mutation. The work
presented suggests that just like in other cancers, BRAF inhibitors in
the clinic might actually promote the growth of those KRAS or NRAS
mutated multiple myeloma clones, reinforcing the need for accurately
identifying the molecularly-based therapeutic approaches in such cases.

The work was conducted as part of the $12 million Multiple Myeloma
Genomics Initiative (MMGI), a progressive genome-mapping program
spearheaded by the MMRF beginning in 2005 to significantly advance the
biological understanding of the disease leading to more precision-based
therapeutic approaches. The analysis was conducted using a combination
of whole-genome sequencing and whole-exome sequencing, performed on
patient samples collected via the MMRC, a network of 16 collaborating
academic medical centers, and processed by centralized tissue bank
housed at the Mayo Scottsdale. All of the data generated through this
project will be made publicly available to cancer researchers worldwide
through the Multiple Myeloma Genomics Portal. Funding for the project
and the portal has been provided by the MMRF.

"The MMRF has played a key, collaborative role in this work. The
Foundation's support and the samples provided by the MMRC enabled the
discoveries highlighted in our paper, and set the stage to advance our
understanding and treatment of multiple myeloma," said co-senior author
Todd Golub, the Broad Institute’s Chief Scientific Officer and Charles
A. Dana Investigator in Human Cancer Genetics at the Dana-Farber Cancer
Institute. Golub is also a professor at Harvard Medical School and an
investigator at Howard Hughes Medical Institute. “This important work
would not have happened without their vision and commitment to advancing
this program over the last eight years.”

To continue the momentum of these study findings, the MMRF has initiated
a series of Precision Medicine initiatives including CoMMpass (Relating Clinical
Outcomes in MM to Personal Assessment
of Genetic Profile), a landmark longitudinal study of 1,000 newly
diagnosed patients with comprehensive molecular profiling and clinical
data collection over the course of the patient’s disease. In 2014, the
MMRF is also planning to initiate a number of clinical trials with
targeted agents in molecularly-selected patients.

About the Multiple Myeloma Genomics Initiative (MMGI)

The MMGI is a $12 million program that comprises several research and
discovery efforts spanning the spectrum of genome science. The MMRC
contributes patient samples from its 16 collaborating academic members
via a centralized Tissue Bank; the MMRF provides funding for the
project. The comprehensive genomic survey of MMRC samples is conducted
in collaboration with the Broad Institute and the Translational Genomics
Research Institute (TGen). The MMGI comprises several research and
discovery efforts spanning the spectrum of genome science. Array
comparative genomic hybridization (aCGH), gene expression profiling
(GEP), DNA methylation analysis and DNA sequencing are among the assays
performed on hundreds of patient multiple myeloma tumor tissue. Data
from the MMGI and other multiple myeloma genomics efforts is available
to the scientific community through the Multiple Myeloma Genomics
Portal, (www.myelomagenomics.org).

About Multiple Myeloma

Multiple myeloma is an incurable blood cancer. The five-year relative
survival rate for multiple myeloma is approximately 43 percent, one of
the lowest of all cancers. In 2013, more than 22,000 adults in the
United States will be diagnosed with multiple myeloma and nearly 11,000
people are predicted to die from the disease.

About the Multiple Myeloma Research Foundation (MMRF)

The Multiple Myeloma Research Foundation (MMRF) was established in 1998
as a 501(c)(3) non-profit organization by twin sisters Karen Andrews and
Kathy Giusti, soon after Kathy's diagnosis with multiple myeloma. The
mission of the MMRF is to relentlessly pursue innovative means that
accelerate the development of next-generation multiple myeloma
treatments to extend the lives of patients and lead to a cure. As the
world's number-one private funder of multiple myeloma research, the MMRF
has raised over $240 million since its inception and directs 90% of
total budget to research and related programming. As a result, the MMRF
has been awarded Charity Navigator’s coveted four-star rating for 11
consecutive years, the highest designation for outstanding fiscal
responsibility and exceptional efficiency. For more information about
the MMRF, please visit: www.themmrf.org.

About the Multiple Myeloma Research Consortium

The Multiple Myeloma Research Consortium (MMRC) is a 509(a)(3)
non-profit organization which integrates leading academic institutions
to accelerate drug development in multiple myeloma. It is led from MMRC
offices in Norwalk, Conn., and comprises 16 member institutions:
University of California, San Francisco, City of Hope, Dana-Farber
Cancer Institute, Emory University's Winship Cancer Institute, the
Cancer Center at Hackensack University Medical Center, H. Lee Moffitt
Cancer Center & Research Institute, Mayo Clinic, Ohio State University,
Mount Sinai School of Medicine, University Health Network (Princess
Margaret Hospital), University of Chicago, University of Michigan,
Washington University, Baylor Charles A. Sammons Cancer Center, Sarah
Cannon Research Institute and Virginia Cancer Specialists. The MMRC was
founded in 2004 by Kathy Giusti, a myeloma patient, and with the help of
the scientific community. The MMRC is a sister organization to the
Multiple Myeloma Research Foundation (MMRF), the world's leading funder
of multiple myeloma research. The MMRC is widely recognized as an
optimal research model to rapidly address critical challenges in drug
development and to explore opportunities in the today's most promising
research areas in genomics, compound validation, and clinical trials.
The MMRC is the only consortium to join academic institutions through
membership agreements, customized IT systems, and an integrated tissue
bank. For more information, please visit www.themmrc.org.